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Alnylam Presents Pre-clinical Data from Hypercholesterolemia, Liver Cancer, Ebola, and Progressive Multifocal Leukoencephalopathy Programs at RNAi Keystone Symposium
Business Wire, March 31, 2008
New Data Highlight Broad Applications of RNAi Therapeutics and Continued Progress in Advancing Pipeline of Innovative Medicines
New Results Also Document Significant Advancements in Systemic Delivery of RNAi Therapeutics
CAMBRIDGE, Mass. -- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it presented pre-clinical data at the "RNAi, MicroRNA, and Non-Coding RNA" Keystone Symposium held March 25-30, 2008 in Whistler, British Columbia. Alnylam and its collaborators presented data from Alnylam's therapeutic programs including hypercholesterolemia, liver cancer, Ebola, and PML, as well as an update on delivery approaches for the systemic delivery of RNAi therapeutics.
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"Alnylam scientists and collaborators continue to make significant progress with RNAi therapeutics in important areas of unmet medical need. We are encouraged by the data continuing to emerge from our hypercholesterolemia, Ebola, liver cancer, and PML programs which show potent and specific in vivo efficacy mediated by an RNAi mechanism," said Victor Kotelianski, M.D., Ph.D., Vice President, Research at Alnylam. "We are also excited by our progress in optimizing siRNAs for systemic delivery, including our recent data regarding heart and muscle delivery. We believe that these cumulative data demonstrate Alnylam's scientific leadership and commitment in translating the science of RNAi into a new class of innovative medicines."
Hypercholesterolemia
Alnylam is developing a systemically delivered RNAi therapeutic for the treatment of hypercholesterolemia targeting PCSK9, a well-validated gene involved in the metabolism of LDL cholesterol ("bad cholesterol"). In a poster titled "RNAi Therapeutics Targeting PCSK9 Acutely Lower Cholesterol from Mice to Non-Human Primates," Alnylam scientists presented in vivo data demonstrating that an RNAi therapeutic targeting the PCSK9 gene significantly decreased LDL cholesterol in three pre-clinical animal models - mouse, rat and non-human primate.
Data presented at the meeting from studies in rodent models include the following:
* an RNAi therapeutic targeting PCSK9 demonstrated statistically significant dose- dependent silencing of PCSK9 mRNA and lowered total cholesterol by 40 to 50 percent as compared with control, non-specific siRNAs; and,
* the effects on cholesterol lowering was mediated by a three to five fold increase in LDL receptor levels with no effect on hepatic triglycerides and hepatic cholesterol; these data further support the belief that RNAi therapeutics targeting PCSK9 are unlikely to result in steatosis (fatty liver).
In non-human primate models, data presented include the following as compared to a control siRNA:
* RNAi-mediated gene silencing was associated with rapid reductions in LDL cholesterol levels by 40 to 60 percent of pre-dose levels;
* circulating apolipoprotein B (apoB) levels - a constituent of the LDL particle - decreased by 30 to 40 percent of pre-dose levels;
* after a single intravenous injection, the RNAi therapeutic showed a durable biological effect with levels of LDL cholesterol decreased for up to three weeks; and,
* therapeutic efficacy was observed with an overall decreased ratio of total cholesterol to HDL cholesterol ("good cholesterol") - a result which has been shown in humans to correlate with clinical benefit.
Liver Cancer
Alnylam is developing a systemically delivered RNAi therapeutic, ALN-VSP, for the treatment of liver cancers and potentially other solid tumors. ALN-VSP comprises two siRNAs in a lipid particle formulation. These two siRNAs target separate genes involved in the growth and development of tumors: kinesin spindle protein, or KSP, and vascular endothelial growth factor, or VEGF. In a talk titled "Translating RNAi," Muthiah Manoharan, Ph.D., Vice President, Drug Discovery at Alnylam presented in vivo data from its ALN-VSP program which were generated in collaboration with Protiva, using their stable nucleic acid-lipid particles, or SNALP, technology.
Pre-clinical data with ALN-VSP, as compared to a control non-specific siRNA, in a mouse model of liver cancer demonstrated the following:
* significant dose-dependent silencing of both KSP and VEGF derived from the human tumor;
* evidence of tumor cell cycle arrest due to KSP silencing documented histologically;
* reduction in overall tumor growth as measured by quantification of a tumor-specific gene; and
* marked reduction in the size of liver tumors in VSP-treated animals as observed by gross pathology.
Alnylam BioDefense(TM)/Ebola
Alnylam is developing an RNAi therapeutic directed against the Ebola virus, which can cause a severe, often fatal infection, and poses a potential biological safety risk and bioterrorism threat. In a poster titled "RNAi Therapeutics for the Treatment of Ebola Virus Infection," pre-clinical data were presented on this program, which utilizes an optimized RNAi therapeutic formulated in a lipid particle for systemic delivery. This work was done in collaboration with Tekmira using their lipid particle delivery formulation technology.
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